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WHIM syndrome 1(WHIMS1)

MedGen UID:
1778124
Concept ID:
C5542296
Disease or Syndrome
Synonym: WHIMS1
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
Not genetically inherited
MedGen UID:
988794
Concept ID:
CN307044
Finding
Source: Orphanet
clinical entity without genetic inheritance.
 
Gene (location): CXCR4 (2q22.1)
 
Monarch Initiative: MONDO:8000006
OMIM®: 193670
Orphanet: ORPHA51636

Definition

WHIM syndrome-1 (WHIMS1) is an autosomal dominant immunologic disorder characterized by neutropenia, hypogammaglobulinemia, and warts due to human papillomavirus (HPV) infection. Despite the peripheral neutropenia, bone marrow aspirates from affected individuals contain abundant mature myeloid cells, a condition termed myelokathexis. The susceptibility to HPV is disproportionate compared with other immunodeficiency conditions (summary by Hernandez et al., 2003). Heusinkveld et al. (2019) provided a detailed review of the clinical features, proposed pathogenesis, and possible therapeutic treatments of WHIM syndrome. There is significant phenotypic variation among patients, such that some individuals may have an 'incomplete' form of the disorder in which one or more of the classic tetrad features are not present. In general, the WHIMS phenotype comprises a spectrum of manifestations with variable expressivity. The pathogenesis of WHIMS1 is postulated to result from impaired CXCL12 (600835)-induced internalization of CXCR4, resulting in prolonged receptor presence at the cell surface that likely contributes to amplification of signaling with a gain-of-function effect. Genetic Heterogeneity of WHIM Syndrome See also WHIMS2 (619407), caused by mutation in the CXCR2 gene (146928) on chromosome 2q35. [from OMIM]

Clinical features

From HPO
Verrucae
MedGen UID:
777120
Concept ID:
C3665596
Finding
Warts, benign growths on the skin or mucous membranes that cause cosmetic problems as well as pain and discomfort. Warts most often occur on the hands, feet, and genital areas.
Abnormal female external genitalia morphology
MedGen UID:
867445
Concept ID:
C4021822
Anatomical Abnormality
Any structural abnormality of the female external genitalia.
Abnormal morphology of female internal genitalia
MedGen UID:
871402
Concept ID:
C4025900
Anatomical Abnormality
An abnormality of the female internal genitalia.
Myelokathexis
MedGen UID:
543867
Concept ID:
C0272173
Disease or Syndrome
Impaired egress of mature neutrophils from bone marrow causing neutropenia.
Bone marrow hypercellularity
MedGen UID:
605828
Concept ID:
C0427703
Finding
A larger than normal amount or percentage of hematopoietic cells relative to marrow fat.
Abnormal bone marrow cell morphology
MedGen UID:
892905
Concept ID:
C4021634
Anatomical Abnormality
An anomaly of the form or number of cells in the bone marrow.
Bronchiectasis
MedGen UID:
14234
Concept ID:
C0006267
Disease or Syndrome
Persistent abnormal dilatation of the bronchi owing to localized and irreversible destruction and widening of the large airways.
Recurrent upper respiratory tract infections
MedGen UID:
154380
Concept ID:
C0581381
Disease or Syndrome
An increased susceptibility to upper respiratory tract infections as manifested by a history of recurrent upper respiratory tract infections (running ears - otitis, sinusitis, pharyngitis, tonsillitis).
Neutropenia
MedGen UID:
163121
Concept ID:
C0853697
Finding
An abnormally low number of neutrophils in the peripheral blood.
Recurrent bacterial infections
MedGen UID:
334943
Concept ID:
C1844383
Finding
Increased susceptibility to bacterial infections, as manifested by recurrent episodes of bacterial infection.
Decreased circulating antibody concentration
MedGen UID:
892481
Concept ID:
C4048270
Finding
An abnormally decreased level of immunoglobulin in blood.
Decreased circulating IgG concentration
MedGen UID:
1720114
Concept ID:
C5234937
Finding
An abnormally decreased level of immunoglobulin G (IgG) in blood.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVWHIM syndrome 1

Professional guidelines

PubMed

Spoor J, Farajifard H, Keshavarz-Fathi M, Rezaei N
J Pediatr Hematol Oncol 2023 Jul 1;45(5):e643-e649. Epub 2023 Apr 10 doi: 10.1097/MPH.0000000000002670. PMID: 37053506
McDermott DH, Murphy PM
Immunol Rev 2019 Jan;287(1):91-102. doi: 10.1111/imr.12719. PMID: 30565238

Recent clinical studies

Etiology

Dobrewa W, Bielska M, Bąbol-Pokora K, Janczar S, Młynarski W
Mutat Res Rev Mutat Res 2024 Jan-Jun;793:108476. Epub 2023 Nov 19 doi: 10.1016/j.mrrev.2023.108476. PMID: 37989463
Bidkhori HR, Bahrami AR, Farshchian M, Heirani-Tabasi A, Mirahmadi M, Hasanzadeh H, Ahmadiankia N, Faridhosseini R, Dastpak M, Shabgah AG, Matin MM
Cell Transplant 2021 Jan-Dec;30:9636897211054498. doi: 10.1177/09636897211054498. PMID: 34807749Free PMC Article
Dale DC, Firkin F, Bolyard AA, Kelley M, Makaryan V, Gorelick KJ, Ebrahim T, Garg V, Tang W, Jiang H, Skerlj R, Beaussant Cohen S
Blood 2020 Dec 24;136(26):2994-3003. doi: 10.1182/blood.2020007197. PMID: 32870250Free PMC Article
Dale DC, Dick E, Kelley M, Makaryan V, Connelly J, Bolyard AA
Curr Opin Hematol 2020 Jan;27(1):11-17. doi: 10.1097/MOH.0000000000000554. PMID: 31652152Free PMC Article
Stiehm ER
Pediatr Res 1993 Jan;33(1 Suppl):S2-7; discussion S7-8. doi: 10.1203/00006450-199305001-00007. PMID: 8433870

Diagnosis

Evans MO 2nd, Petersen MM, Khojah A, Jyonouchi SC, Edwardson GS, Khan YW, Connelly JA, Morris D, Majumdar S, McDermott DH, Walter JE, Murphy PM
J Clin Immunol 2021 Apr;41(3):621-628. Epub 2021 Jan 7 doi: 10.1007/s10875-020-00921-4. PMID: 33415666Free PMC Article
Heusinkveld LE, Majumdar S, Gao JL, McDermott DH, Murphy PM
J Clin Immunol 2019 Aug;39(6):532-556. Epub 2019 Jul 16 doi: 10.1007/s10875-019-00665-w. PMID: 31313072Free PMC Article
Majumdar S, Murphy PM
Int J Mol Sci 2018 Dec 20;20(1) doi: 10.3390/ijms20010003. PMID: 30577453Free PMC Article
Al Ustwani O, Kurzrock R, Wetzler M
Br J Haematol 2014 Jan;164(1):15-23. Epub 2013 Sep 20 doi: 10.1111/bjh.12574. PMID: 24111611Free PMC Article
Dotta L, Tassone L, Badolato R
Curr Mol Med 2011 Jun;11(4):317-25. doi: 10.2174/156652411795677963. PMID: 21506920

Therapy

Hoy SM
Drugs 2024 Aug;84(8):969-975. Epub 2024 Jul 15 doi: 10.1007/s40265-024-02063-y. PMID: 39004659
Badolato R, Alsina L, Azar A, Bertrand Y, Bolyard AA, Dale D, Deyà-Martínez À, Dickerson KE, Ezra N, Hasle H, Kang HJ, Kiani-Alikhan S, Kuijpers TW, Kulagin A, Langguth D, Levin C, Neth O, Olbrich P, Peake J, Rodina Y, Rutten CE, Shcherbina A, Tarrant TK, Vossen MG, Wysocki CA, Belschner A, Bridger GJ, Chen K, Dubuc S, Hu Y, Jiang H, Li S, MacLeod R, Stewart M, Taveras AG, Yan T, Donadieu J
Blood 2024 Jul 4;144(1):35-45. doi: 10.1182/blood.2023022658. PMID: 38643510Free PMC Article
Evans MO 2nd, Petersen MM, Khojah A, Jyonouchi SC, Edwardson GS, Khan YW, Connelly JA, Morris D, Majumdar S, McDermott DH, Walter JE, Murphy PM
J Clin Immunol 2021 Apr;41(3):621-628. Epub 2021 Jan 7 doi: 10.1007/s10875-020-00921-4. PMID: 33415666Free PMC Article
Al Ustwani O, Kurzrock R, Wetzler M
Br J Haematol 2014 Jan;164(1):15-23. Epub 2013 Sep 20 doi: 10.1111/bjh.12574. PMID: 24111611Free PMC Article
Debnath B, Xu S, Grande F, Garofalo A, Neamati N
Theranostics 2013;3(1):47-75. Epub 2013 Jan 15 doi: 10.7150/thno.5376. PMID: 23382786Free PMC Article

Prognosis

Dobrewa W, Bielska M, Bąbol-Pokora K, Janczar S, Młynarski W
Mutat Res Rev Mutat Res 2024 Jan-Jun;793:108476. Epub 2023 Nov 19 doi: 10.1016/j.mrrev.2023.108476. PMID: 37989463
Spoor J, Farajifard H, Keshavarz-Fathi M, Rezaei N
J Pediatr Hematol Oncol 2023 Jul 1;45(5):e643-e649. Epub 2023 Apr 10 doi: 10.1097/MPH.0000000000002670. PMID: 37053506
Ma X, Wang Y, Wu P, Kang M, Hong Y, Xue Y, Chen C, Li H, Fang Y
Front Immunol 2022;13:857527. Epub 2022 Apr 13 doi: 10.3389/fimmu.2022.857527. PMID: 35493524Free PMC Article
Laberko A, Deordieva E, Krivan G, Goda V, Bhar S, Kawahara Y, Rao K, Worth A, McDermott DH, Balashov D, Maschan A, Shcherbina A
J Clin Immunol 2022 Jan;42(1):171-182. Epub 2021 Oct 26 doi: 10.1007/s10875-021-01155-8. PMID: 34697698Free PMC Article
Imashuku S, Miyagawa A, Chiyonobu T, Ishida H, Yoshihara T, Teramura T, Kuriyama K, Imamura T, Hibi S, Morimoto A, Todo S
Ann Hematol 2002 Aug;81(8):470-3. Epub 2002 Jul 20 doi: 10.1007/s00277-002-0489-9. PMID: 12224006

Clinical prediction guides

Hoy SM
Drugs 2024 Aug;84(8):969-975. Epub 2024 Jul 15 doi: 10.1007/s40265-024-02063-y. PMID: 39004659
Badolato R, Alsina L, Azar A, Bertrand Y, Bolyard AA, Dale D, Deyà-Martínez À, Dickerson KE, Ezra N, Hasle H, Kang HJ, Kiani-Alikhan S, Kuijpers TW, Kulagin A, Langguth D, Levin C, Neth O, Olbrich P, Peake J, Rodina Y, Rutten CE, Shcherbina A, Tarrant TK, Vossen MG, Wysocki CA, Belschner A, Bridger GJ, Chen K, Dubuc S, Hu Y, Jiang H, Li S, MacLeod R, Stewart M, Taveras AG, Yan T, Donadieu J
Blood 2024 Jul 4;144(1):35-45. doi: 10.1182/blood.2023022658. PMID: 38643510Free PMC Article
Anginot A, Nguyen J, Abou Nader Z, Rondeau V, Bonaud A, Kalogeraki M, Boutin A, Lemos JP, Bisio V, Koenen J, Hanna Doumit Sakr L, Picart A, Coudert A, Provot S, Dulphy N, Aurrand-Lions M, Mancini SJC, Lazennec G, McDermott DH, Guidez F, Blin-Wakkach C, Murphy PM, Cohen-Solal M, Espéli M, Rouleau M, Balabanian K
Nat Commun 2023 Apr 12;14(1):2058. doi: 10.1038/s41467-023-37791-4. PMID: 37045841Free PMC Article
Geier CB, Ellison M, Cruz R, Pawar S, Leiss-Piller A, Zmajkovicova K, McNulty SM, Yilmaz M, Evans MO 2nd, Gordon S, Ujhazi B, Wiest I, Abolhassani H, Aghamohammadi A, Barmettler S, Bhar S, Bondarenko A, Bolyard AA, Buchbinder D, Cada M, Cavieres M, Connelly JA, Dale DC, Deordieva E, Dorsey MJ, Drysdale SB, Ehl S, Elfeky R, Fioredda F, Firkin F, Förster-Waldl E, Geng B, Goda V, Gonzalez-Granado L, Grunebaum E, Grzesk E, Henrickson SE, Hilfanova A, Hiwatari M, Imai C, Ip W, Jyonouchi S, Kanegane H, Kawahara Y, Khojah AM, Kim VH, Kojić M, Kołtan S, Krivan G, Langguth D, Lau YL, Leung D, Miano M, Mersyanova I, Mousallem T, Muskat M, Naoum FA, Noronha SA, Ouederni M, Ozono S, Richmond GW, Sakovich I, Salzer U, Schuetz C, Seeborg FO, Sharapova SO, Sockel K, Volokha A, von Bonin M, Warnatz K, Wegehaupt O, Weinberg GA, Wong KJ, Worth A, Yu H, Zharankova Y, Zhao X, Devlin L, Badarau A, Csomos K, Keszei M, Pereira J, Taveras AG, Beaussant-Cohen SL, Ong MS, Shcherbina A, Walter JE
J Clin Immunol 2022 Nov;42(8):1748-1765. Epub 2022 Aug 10 doi: 10.1007/s10875-022-01312-7. PMID: 35947323Free PMC Article
Stiehm ER
Pediatr Res 1993 Jan;33(1 Suppl):S2-7; discussion S7-8. doi: 10.1203/00006450-199305001-00007. PMID: 8433870

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